Study of Ultomiris® (Ravulizumab) Safety in Pregnancy

Alexion Pharmaceuticals

Status: Enrolling

Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH); Ultomiris-exposed Pregnant/ Postpartum; Neuromyelitis Optica Spectrum Disorder (NMOSD); Generalized Myasthenia Gravis (gMG); Atypical Hemolytic Uremic Syndrome (aHUS); Pregnancy

Treatments: Drug: Ultomiris

Study type: Observational

Funder types: Industry

Identifiers

NCT06312644

D9289C00007

Details and patient eligibility

About

The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of selected fetal/neonatal/infant outcomes in utero, at birth, and through 1 year of age after exposure in utero or via breastmilk.

Full description

This observational study will collect prospective and retrospective data in participants exposed to Ultomiris during pregnancy and/or breastfeeding to assess risk of maternal complications, adverse effects on the developing fetus, and adverse effects on the infant.

Enrollment: 75 estimated patients

Sex: Female

Volunteers: No Healthy Volunteers

Inclusion criteria

• Female participant must have a medically confirmed qualifying pregnancy (prospectively or retrospectively identified).
• Participant informed consent (written or e-consent per local regulations or ethics committee requirements) must be obtained prior to the participant's enrollment. If the participant is a minor, consent must be obtained from the parent or legal guardian, with assent from the minor (as locally appropriate).
• Willing to provide contact information for the participant.
• Willing to authorize HCP(s) to release maternal and infant medical information to the study, upon request, if applicable to current local regulations.
• Diagnosed with an indication for which Ultomiris is approved, based on HCP or medical records.
• Exposed to Ultomiris at any point during the defined exposure window based on HCP or medical record documentation. (If exact exposure dates are unknown, the reporter must be able to specify or estimate trimester or timing of exposure [prior to conception as LMP+14 days, or during breastfeeding].)
• Use of Ultomiris per local product information (i.e., United States Prescribing Information [USPI] or summary of product characteristics [SmPC])

Exclusion criteria

• Participants who are unable to provide consent or assent (as locally appropriate) (e.g., diagnosed with severe psychiatric conditions or severe intellectual disabilities) will be excluded from this study